Newer advances in the treatment of Duchenne muscular dystrophy and spinal muscular atrophy

Abstract

Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) are two common and important Inherited neuromuscular disorders which have witnessed immense advances in their treatment owing to ongoing developments in gene therapy. Better modalities for clinical testing and improved clinical awareness has led to facilitation of innovative therapeutic research. Multiple new agents have been approved by regulatory authorities. A continuing research on evaluating such treatment options is required more than ever. These novel therapies have immense potential to transform this field and prolong the functional independence and lifespan of patients.

Keywords

• duchenne muscular dystrophy
• exon skipping
• nusinersen
• recent advances
• risdiplam
• spinal muscular atrophy
• vamorolone
• zolgensma